Treatment of Inherited and Genetic Cardiomyopathies by ERK Inhibition
Treatment of Inherited and Genetic Cardiomyopathies by ERK Inhibition
This technology discovered that genes related to the ERK and JNK pathways exhibited statistically different expression at the incipience of the development of cardiomyopathies. Treatment with ERK inhibitor alone or treatment with both JNK inhibitor and ERK inhibitor significantly improved cardiac function assessed by echocardiography in EDMD mice. Activation of ERK or ERK plus JNK can lead to heart disease in EDMD and other inherited ca...
New York, NY, United States
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Background

Treatment for Emery-Dreifuss Muscular Dystrophy (EDMD) Emery-Dreifuss Muscular Dystrophy (EDMD) is a form of genetic, degenerative muscular dystrophy disease affecting muscles used for movement (skeletal muscles) and heart (cardiac) muscle. The major cause of mortality and morbidity in EDMD is cardiomyopathy. So far, no specific treatment for EDMD exists. While EDMD is a relatively uncommon disorder, mutations in the same gene (LMNA) that cause autosomal dominant EDMD may in fact be responsible for 8% or inherited and sporadic dilated cardiomyopathies. While pacemakers and defibrillators may be useful in preventing sudden death in these patients, worsening heart failure invariably occurs until there is not curative intervention short of heart transplantation. This technology identified signaling pathways involved in the development of cardiomyopathy in a mouse model of autosomal dominant and X-linked EDMD. Furthermore, the technology showed the inhibition of these signaling pathways could improve cardiac functions in an animal model of autosomal EDMD, which can be used as novel treatments for EDMD. 

Treating EDMD with ERK Inhibitor or ERK and JNK Inhibitors This technology discovered that genes related to the ERK and JNK pathways exhibited statistically different expression at the incipience of the development of cardiomyopathies. Treatment with ERK inhibitor alone or treatment with both JNK inhibitor and ERK inhibitor significantly improved cardiac function assessed by echocardiography in EDMD mice. Activation of ERK or ERK plus JNK can lead to heart disease in EDMD and other inherited cardiomyopathies. 

Applications: • The technology provides novel treatments for EDMD and other cardiomyopathies caused by mutation in the LMNA gene by targeting ERK and JNK pathway • The technology can be also used as potential treatments for other inherited forms of cardiomyopathies • The signaling pathway identified by this technology can be used to develop diagnostic and screening assay to test the efficacy of drugs in treatments for cardiomyopathies 

Advantages: The technology presents a novel treatment for cardiomyopathy caused by LMNA mutation, and possible other cardiomyopathies, which cannot be achieved by any alternative methods yet. 

Publications: • Muchir et al. Hum Mol Genet. 2007 Aug 1;16(15):1884-95. • Muchir et al. J Clin Invest. 2007 May;117(5):1282-93. • Muchir et al. Hum Mol Genet. 2009 Jan 15;18(2):241-7

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